The World Organization for Research on Lysosomal Diseases (WORLD) Clinical Research Center at the University of Minnesota will host the first annual symposium on lysosomal diseases (LD), May 13-15, 2004, in Minneapolis, Minnesota. This symposium is organized to provide an annual multidisciplinary forum to facilitate discussion of basic/clinical research and care issues in LD. Participants include clinicians, geneticists/genetic counselors, neurologists/neuropsychologists, researchers, health care professionals, patients/families, support organizations and industry professionals. Specific aims are to: 1) foster interdisciplinary collaboration with the overall goal of improving knowledge of basic discoveries and clinical manifestations of these diseases; 2) identify and discuss the latest findings in the natural history of LD, diagnostic testing and screening, and treatment; and 3) identify areas requiring additional basic/clinical research. The 2004 meeting is organized into 4 symposium topics, which will include a combination of expert invited speakers, platform presentations selected from submitted abstracts and panel discussions. The Symposium on Longitudinal and Quality of Life (QOL) Studies will identify critical elements in designing and assessing longitudinal and QOL studies for LD. The Symposium on Bioethics and Regulatory Issues will identify and discuss bioethical issues that impact rare disease clinical research. A Symposium on Innovative Therapies will provide an understanding of the pathophysiology and molecular biology of LD and will present new diagnostic techniques/treatment advances, with a focused session on advances in hematopoietic cell transplantation and enzyme replacement therapy. The Symposium of Excellence on Mucopolysaccharidosis: Airway Safety First will identify the role of disease and patient-specific factors in pre-, peri-, and post-operative assessment and management, and pulmonary and respiratory conditions that impact the health and well being of MPS patients. Patients/family sessions will include educational presentations, support services, and round table discussions. [unreadable] [unreadable]